Reshma Kewalramani, Vertex

Ver­tex is poised for a shop­ping spree. But it’s still un­clear just what might go in­to their bas­ket — and at least one an­a­lyst is get­ting im­pa­tient

Af­ter set­ting the tone at JP Mor­gan for a busy year of deal­mak­ing, Resh­ma Ke­wal­ra­mani and her Ver­tex crew are giv­ing slight­ly more shape to the pipeline they want to build with $6.7 bil­lion in cash — but leav­ing much to be spec­u­lat­ed.

Through it all, the fresh­man CEO says, Ver­tex will re­main “a com­pa­ny that is about spe­cial­ty mar­kets” — and em­phat­i­cal­ly not a rare dis­ease play­er.

Ge­off Porges at SVB Leerink, though, sees the dri­ve to in­tro­duce new drugs to the pipeline more dri­ven by ne­ces­si­ty than any­thing else. The com­pa­ny, ac­cord­ing to him, is avoid­ing key ques­tions while ex­pos­ing “in­con­sis­ten­cies be­tween their port­fo­lio and their strat­e­gy.”

“(T)he com­pa­ny’s suc­cess is catch­ing up with them, in terms of the bur­den of hav­ing to di­ver­si­fy their busi­ness and add to their pipeline,” he wrote in a note. “Notwith­stand­ing the com­pa­ny’s catch­phras­es of ‘crack­ing the bi­ol­o­gy’ and ‘pour­ing on the chem­istry’, their most ad­vanced pro­gram is CTX-001 which is an al­lo­gene­ic stem ther­a­py treat­ment for sick­le cell dis­ease and be­ta tha­lassemia. An­oth­er of their hand­ful of clin­i­cal pro­grams is their pan­cre­at­ic islet cell trans­plant ther­a­py for type I di­a­betes (en­ter­ing clin­ic 1H 21).”

Rather than specifics, she of­fered some “col­or and tex­ture” be­hind cap­i­tal al­lo­ca­tion: Com­pared to three years ago, Ver­tex mol­e­cules are in pa­tients across six dis­ease ar­eas (up from just two, cys­tic fi­bro­sis and pain) and rev­enue has grown from a lit­tle north of $2 bil­lion to more than $6 bil­lion.

As she sug­gest­ed at JPM, it all backs up a search for mid-to late-stage as­sets along­side new tools:

I want to be very clear that the strat­e­gy is ex­act­ly the same what I out­lined pre­vi­ous­ly. And if I just fo­cus in on the as­sets that fit our R&D strat­e­gy, we are now able to look at, for ex­am­ple, Phase 2 as­sets, as­sets that might be in Phase 3, and those are as­sets that we’re go­ing to look at. We’re al­so go­ing to con­tin­ue to look at tools for our toolk­it. I’m not look­ing at — I have no pre­con­ceived no­tions about the tim­ing of a trans­ac­tion and I have no pre­con­ceived no­tions about the dol­lar amount of a trans­ac­tion. It has to fit our R&D strat­e­gy. It has to be trans­for­ma­tive. We have to be able to add val­ue. And, when we find that as­set, and we have the pa­tience and the judg­ment to be very thought­ful about that, we’re go­ing to be ready to eval­u­ate.

David Alt­shuler

The ex­ecs al­so dodged a ques­tion on whether there is any plat­form tech that they don’t al­ready have but would be in­ter­est­ed in.

They have al­ready dipped their toes in­to some of the hottest pools, in­clud­ing gene ther­a­py, gene edit­ing, mR­NA, pro­tein degra­da­tion and small mol­e­cule ap­proach­es for drug­ging RNA. “And you will con­tin­ue to see us do deals and part­ner­ships where there’s a par­tic­u­lar tech­nol­o­gy that opens up a tar­get that we think has trans­for­ma­tion­al po­ten­tial, and we’ll con­tin­ue to do those as time goes on,” was as far as R&D chief David Alt­shuler was will­ing to go.

For now, at least.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

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Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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